A Study to Collect Information of People With Narcolepsy in Spain (SOMNUS)

Observational, Multicentre, Cross-sectional Study to Describe Diagnosis and Treatment Patterns in Narcolepsy Patients in Real Life Practice in Spain

This study aims to review information of people with a sleep condition called narcolepsy. Narcolepsy is a condition that causes extreme sleepiness during the day including falling asleep suddenly. Study doctors will review the medical records of the participants from sleep clinics in Spain. They will do this from 1 year before the participant was diagnosed with narcolepsy up to the start of this study.

Participants will visit the sleep clinic once. During this visit, the study doctors will carry out a short medical exam. Participants will also complete a few questionnaires during this visit. If participants cannot visit the clinic for any reason, the clinic staff will arrange a phone call instead.

Study Overview

• Status: Completed
• Conditions: Narcolepsy

Detailed Description

This is a non-interventional, cross-sectional study with retrospective medical chart review conducted in public and private Spanish sleep clinics, to describe the management of adult participants diagnosed with narcolepsy defined by ICDS-3 in real world practice.

This study will enroll approximately 196 participants (137 with NT1 and 59 with NT2). Participants will be enrolled in 2 cohorts:

• Cohort A: NT1 Participants
• Cohort B: NT2 Participants

The data will be collected retrospectively through clinical records and at study visit (clinical and demographic data, and participant related outcomes [PRO]) in an electronic case report form (eCRF).

This multi-center study will be conducted in Spain. The overall duration of this study is approximately 24 months. Participants will attend a single visit in the participating centers. In the event of a possible outbreak of coronavirus disease 2019 (COVID-19) participants will have a telepathic visits, the invitation to participate, the obtention of participant's informed consent and the collection of participant's questionnaires will be carried out remotely (by phone or via email).

• Study Type: Observational
• Enrollment (Actual): 214

Contacts and Locations

Study Locations

• Spain
  • Cataluna
    • Barcelona, Cataluna, Spain, 8036
      • Hospital Clinic de Barcelona

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with confirmed diagnosis of narcolepsy defined by ICDS-3.

Description

Inclusion Criteria:

1. With confirmed diagnosis of narcolepsy defined by ICDS-3.
2. With at least 1-year follow-up with data available at the participating site after initial narcolepsy diagnosis and before study inclusion.
3. With data available at the participant site at least 1-year before first narcolepsy diagnosis.
4. Capable to fulfill the study questionnaires.

Exclusion Criteria:

1. Participating in a clinical trial (less than or equal to [<=] 12 months).
2. With any serious degenerative disease (Alzheimer, Parkinson or epilepsy) or psychiatric condition.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
Narcolepsy Type 1; Participants with confirmed diagnosis of narcolepsy type 1 (NT1) defined by the International Classification of Sleep Disorders, Third Edition (ICDS-3) will be observed for up to 16 months.
Narcolepsy Type 2; Participants with confirmed diagnosis of narcolepsy type 2 (NT2) defined by the ICDS-3 will be observed for up to 16 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants Categorized Based on Treatment by Narcolepsy Type Since Diagnosis up to Study Visit	Percentage of participants with narcolepsy categorized based on pharmacological and non-pharmacological treatment as 'yes' and 'no' since study diagnosis are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point.	From narcolepsy diagnosis up to study visit (up to 12 months)
Percentage of Participants Categorized Based on Non-Pharmacological Treatments Patterns by Treatment Status	Percentage of participants with narcolepsy categorized based on non-pharmacological treatment patterns as 'yes' and 'no' by treatment status [Naïve (untreated) and treated] are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point. Data is collected and reported per Naïve (untreated) and treated arms for this outcome measure.	From narcolepsy diagnosis up to study visit (up to 12 months)
Percentage of Treated or Naïve (Untreated) Participants for the Type of Pharmacological Treatment Status Since Diagnosis	Percentage of Treated or naïve (Untreated) participants with narcolepsy categorized based on pharmacological treatment status since diagnosis are reported in this outcome measure. Percentages are rounded-off to the nearest decimal point.	From narcolepsy diagnosis up to study visit (up to 12 months)
Percentage of Participants With the Use of Each Type of Treatment Stratified by Type of Hospital (Public or Private Centers)	Percentages are rounded off to the nearest decimal point.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants Categorized by Sociodemographic and Clinical Variables at Study Visit	Sociodemographic variables categorized by age, gender (male/female), ethnicity, educational level ongoing or completed level of education (without studies, primary studies, secondary studies, university studies, other superior studies, not available); occupational status and occupation (student, employed, self-employed, employed but on sick leave due to the study disease, permanent incapacity to work due to the study disease, permanent incapacity to work due to other reasons, unemployed, retired, domestic work, other); civil status (married/with partner, divorced/separated, unmarried, widow/er, unknown); living conditions (alone, wife/husband and/or sons, family caregivers, other caregivers, unknown). Clinical variables categorized by smoking status, alcohol intake, exercise status and family history of narcolepsy in first-degree and second-degree relatives].	Day 1
Height of Participants at Study Visit		Day 1
Weight of Participants at Study Visit		Day 1
Body Mass Index (BMI) of Participants at Study Visit	BMI = weight(kg)/height(m)^2	Day 1
Blood Pressure (BP) of Participants at Study Visit		Day 1
Percentage of Participants Categorized Based on Different Specialists Who Diagnosed Narcolepsy	Specialist who diagnosed narcolepsy were categorized as general practitioners, neurologist, neuropediatrician, neurophysiologist, pneumologist, somnologist, somnologist-unit and specialist (Not specified). Percentage of participants are rounded-off to the nearest decimal point.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Percentage of Participants Categorized Based on Usage of Different Procedures or Tests for the Diagnosis of Narcolepsy	Different procedures or tests for diagnosis included clinical history, Clinical Assessment: Epworth sleepiness scale (ESS) [ESS consists of 8 items, on a 4-point scale (0-3) ranging from "0=No chance of dozing" to "3=High chance of dozing" while engaged in eight different activities. The score can range from 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are within the normal range], Neurological Assessment, Multiple sleep latency test (MSLT), Apnea-hypopnea index (AHI), Other procedures, Human leucocyte antigen (HLA) typing, Hypocretin-1 cerebrospinal fluid (CSF) or Orexin. Percentages are rounded-off to the nearest decimal point.	From a year before narcolepsy diagnosis up to 12 months
Time From First Symptom to Diagnosis of Narcolepsy	Time from first symptom in participant to diagnosis is the time when the first symptom of narcolepsy was identified until its diagnosis during retrospective visit. As the first symptom and the diagnosis at retrospective visit could be any time in the past up to end of enrolment in this study. Thus, as pre-planned data for this outcome measure may exceed the duration of the study.	From first symptom of narcolepsy up to diagnosis maximum up to 59.92 years (retrospective data was retrieved and observed during 20 months of this study)
Time From Diagnosis of Narcolepsy to First Treatment	Time from diagnosis to first treatment is the time required to diagnose the first symptom of narcolepsy until the first treatment is provided after diagnosis retrospectively. As the first symptom and the diagnosis at retrospective visit could be any time in the past up to end of enrolment in this study. Thus, as pre-planned data for this outcome measure may exceed the duration of the study.	From first symptom of narcolepsy up to diagnosis maximum up to 46.04 years (retrospective data was retrieved and observed during 20 months of this study)
Percentage of Participants Categorized Based on Pharmacological Treatment and Interventions Received in Relation With the Presence of Most Typical Symptoms		From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Percentage of Participants With Pharmacological Treatments at Each Line	Pharmacological Treatments were categorized as 1st treatment after diagnosis (monotherapy and combinations), treatments used from diagnosis to study visit and treatments at study visit (monotherapy and combinations). Percentages are rounded off to the nearest decimal point.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Sustained Attention to Response Task (SART)	Behavioral measure of working memory, attention, and impulse/inhibitory control - The Sustained Attention to Response Task (SART) is a computer-based go/no-go task that requires participants to withhold behavioral response to a single, infrequent target (often the digit 3) presented amongst a background of frequent non-targets (0-2, 4-9). After each block, two probe questions are presented in succession. The first asks, "Where was your attention focused during this block of trials?" Participants respond on a 6-point Likert scale, where 1 represents, "on task," and 6, "off task." A second question asks, "How aware were you of where your attention was during this block of trials?" Participants respond on a similar scale, where 1 represents, "aware," and 6, "unaware."	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Maintenance of Wakefulness Test (MWT)	The MWT is a validated objective measure that evaluates a person's ability to remain awake under soporific conditions for a defined period of time. This tendency to fall asleep is measured via electroencephalography-derived sleep latency. Sleep onset is defined as the first epoch of greater than 15 seconds of cumulative sleep in a 30-second epoch. During each MWT session (1 session = 40 minutes), participants will be instructed to sit quietly and remain awake for as long as possible. If no sleep has been observed according to these rules, then the latency is defined as 40 minutes. MWT sleep latency ranges from 0 to 40 minutes, with higher scores indicating greater ability to stay awake.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Epworth Sleepiness Scale (ESS)	ESS is a subjective, self-administered questionnaire to assess daytime sleepiness and consists of 8 items, on a 4-point scale (0-3) ranging from "No chance of dozing" to "High chance of dozing" while engaged in eight different activities. The score can range from 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are considered to be within the normal range.	From a year before narcolepsy diagnosis up to 12 months
Percentage of Participants Categorized Based on Different Non-pharmacological Treatment Approach	Non-pharmacological treatment approach was categorized as take short naps, maintain a regular sleep schedule, avoid caffeine or alcohol before bedtime, avoid smoking, especially at night, exercise daily, avoid large, heavy meals right before bedtime, and other. Percentage of participants are rounded-off to the nearest decimal point.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Percentage of Participants Categorized Based on Utilization of Direct Healthcare Resource Utilized Per Year	Direct healthcare resource utilized per year were categorized as treatments for narcolepsy, routine monitoring visits, tests, emergency visits, hospitalizations and complications associated to narcolepsy. Percentage of participants are rounded-off to the nearest decimal point.	From a year before study visit (up to 12 months)
Duration of Hospitalization	Duration of hospitalization is the total number of days at which participants were admitted in hospital.	From a year before study visit (up to 12 months)
Work Productivity and Activity Impairment (WPAI) Score	The WPAI is a questionnaire that assesses the impact of an intervention on work productivity, evaluating 4 areas including absenteeism [Q2/(Q2+Q4)], presenteeism [Q5/10], work productivity loss {Q2/(Q2+Q4)+[(1-(Q2/(Q2+Q4))x(Q5/10)]}, and activity impairment [Q6/10], calculated based on three items: (Q2) the number of hours missed from work due to health problems in the past seven days; (Q4) the number of actual work hours in the past seven days; and (Q5) to what degree did the disease impair the productivity while working past seven days. The data calculated for each area is then converted to percent. Each of the areas is scored separately as a percentage, ranging from 0 to 100, with higher scores indicating greater impairment and less productivity.	From a year before study visit (up to 20 months)
Number of Participants Categorized Based on Occupational Accidents		From a year before study visit (up to 20 months)
Percentage of Participants Categorized Based on Cost of Direct Healthcare Resources Utilized by Participants Per Year	Cost of direct healthcare resources utilized by participants per year based on costs of following direct healthcare resources was to be included: treatment received, routine monitoring visits (specialists and GPs), tests performed, emergency rooms visits, hospitalizations and complications derived from narcolepsy, and unitary costs.	From a year before study visit (approximately 20 months)
Percentage of Participants With Health Related Quality of Life (HRQoL) Assessed by European Quality of Life Five Dimension (EQ-5D) Questionnaire	The EQ-5D questionnaire is a generic instrument for describing and valuing health. It is based on a descriptive system that defines health in terms of 5 dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression. Each dimension has 3 response categories corresponding to no problems, some problems, and extreme problems. The instrument is designed for self-completion, and respondents also rate their overall health on the day of the interview on a 0-100 hash-marked, vertical visual analogue scale (EQ-VAS). Higher score indicates better health state. Percentage of participants are rounded-off to the nearest decimal point.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Stigma Scale for Chronic Illness 8-item Version (SSCI-8) Total Score	SSCl-8 is an 8 items scale (2 items measuring internalized stigma, 5 items measuring enacted stigma, and 1 item, which exhibited split loading, measuring either or both internalized and enacted stigma) developed to assess internalized and enacted/experienced stigma across neurological conditions. It uses a 5-point Likert scale ranging from 1 (never) to 5 (always) for scoring each item. The total score is calculated by summing individual items, ranging from 8 to 40. Higher scores indicate worsening of condition.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Treatment Satisfaction of Participants Assessed by Treatment Satisfaction Questionnaire for Medication (TSQM-9)	TSQM-9 measures participant satisfaction with the medication in 3 domains: Effectiveness, convenience, and global satisfaction. The scores were computed by adding items for each domain, i.e., 1 to 3 for effectiveness, 4 to 6 for convenience, and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) x 3 items = 18 for effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. TSQM-9 domain scores range from 0 to 100, with higher scores indicating greater satisfaction for that domain.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Percentages of Participants Based on Most Prevalent Comorbidities and Other Concomitant Disorders Associated With Narcolepsy	Percentage of participants are rounded-off to the nearest decimal point.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Charlson Comorbidity Index (CCI)	CCI is a scale to estimate 10-year mortality based on a score from a range of 12 comorbidities, the comorbidity score ranges from 0 to a maximum of 24 points. Higher scores indicate the higher chances of mortality.	From a year before narcolepsy diagnosis up to study visit (up to 20 months)
Number of Participants With Adverse Events (AEs)	An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, a new disease or worsening in severity or frequency of a concomitant disease, temporally associated with the use of a medicinal product, whether or not the event is considered causally related to the use of the product.	Up to 20 months

Collaborators and Investigators

• Sponsor: Takeda
• Investigators: Study Director: Study Director, Takeda

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): February 18, 2021
• Primary Completion (Actual): November 4, 2022
• Study Completion (Actual): November 4, 2022

Study Registration Dates

• First Submitted: December 9, 2020
• First Submitted That Met QC Criteria: December 9, 2020
• First Posted (Actual): December 14, 2020

Study Record Updates

• Last Update Posted (Actual): October 4, 2024
• Last Update Submitted That Met QC Criteria: July 3, 2024
• Last Verified: July 1, 2024

More Information

Terms related to this study

• Keywords: Drug Therapy
• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Disorders of Excessive Somnolence; Narcolepsy
• Other Study ID Numbers: TAK-994-5001; U1111-1259-8720 (Registry Identifier: WHO)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• IPD Sharing Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No